Alnylam Clinical Development Pipeline
TRANSTHYRETIN AMYLOIDOSIS (TTR)
PRODUCT
DISEASE
PHASE 1
PHASE 2
PHASE 3
COMMERCIAL
| ONPATTRO® (patisiran) | hATTR Amyloidosis with Polyneuropathy |  |
|
| AMVUTTRA® (vutrisiran) | ATTR Amyloidosis with Cardiomyopathy and hATTR Amyloidosis with Polyneuropathy | |
|
| Nucresiran | ATTR Amyloidosis with Cardiomyopathy | |
|
| Nucresiran | hATTR Amyloidosis with Polyneuropathy | |
PRODUCT
DISEASE
PHASE 1
PHASE 2
PHASE 3
COMMERCIAL
| Leqvio® (inclisiran) | Hypercholesterolemia1 | ||
| Zilebesiran | Hypertension2 | |
|
| Zilebesiran REVERSIR | Hypertension2 | |
PRODUCT
DISEASE
PHASE 1
PHASE 2
PHASE 3
COMMERCIAL
| ALN-4324 (GRB14) | Type 2 Diabetes Mellitus | |
|
| Rapirosiran (ALN-HSD) | Metabolic Dysfunction-Associated Steatohepatitis (MASH)1 | ||
| ALN-PNP | Metabolic Dysfunction-Associated Steatohepatitis (MASH)1 | ||
| ALN-CIDEB | Metabolic Dysfunction-Associated Steatohepatitis (MASH)1 | ||
| ALN-ANG3 | Diabetic Kidney Disease1 | ||
| ALN-2232 (ACVR1C) | Obesity & Weight Management | |
|
| ALN-6222 (INHBE) | Obesity & Weight Management | |
|
| ALN-APOC3 | Dyslipidemia1 |
PRODUCT
DISEASE
PHASE 1
PHASE 2
PHASE 3
COMMERCIAL
| Cemdisiran(+/- Pozelimab) | Myasthenia Gravis1 | ||
| Mivelsiran (ALN-APP) | Cerebral Amyloid Angiopathy (CAA) | |
|
| Mivelsiran (ALN-APP) | Alzheimer’s Disease | |
|
| ALN-SOD | SOD1 Amyotrophic Lateral Sclerosis (ALS)3 | |
|
| Brezosiran (ALN-HTT02) | Huntington’s Disease4 | |
|
| ALN-5288 (MAPT) | Alzheimer’s Disease4 | |
|
| ALN-SNCA | Parkinson’s Disease1 |
PRODUCT
DISEASE
PHASE 1
PHASE 2
PHASE 3
COMMERCIAL
| Qfitlia™ (fitusiran) | Hemophilia A or B1 | ||
| Cemdisiran (+Pozelimab) | Paroxysmal Nocturnal Hemoglobinuria1 | ||
| ALN-6400 (PLG) | Hereditary Hemorrhagic Telangiectasia | |
|
| ALN-6400 (PLG) | Von Willebrand Disease | |
|
| AG-236(ALN-TMP) | Polycythemia Vera1 | ||
| ALN-CFB | Paroxysmal Nocturnal Hemoglobinuria1 |
PRODUCT
DISEASE
PHASE 1
PHASE 2
PHASE 3
COMMERCIAL
| GIVLAARI® (givosiran) | Acute Hepatic Porphyria (AHP) | |
|
| OXLUMO® (lumasiran) | Primary Hyperoxaluria Type 1 (PH1) | |
|
| Cemdisiran (+/- Pozelimab) | Geographic Atrophy1 | ||
| Elebsiran (+Tobevibart) | Hepatitis D Virus Infection1 | ||
| ALN-BCAT | Hepatocellular Carcinoma | |
|
| ALN-4285 | Healthy Volunteers | |
|
| ALN-4915 | Healthy Volunteers | |
|
| ALN-F1202 | Healthy Volunteers1 |
1 Out-licensed with milestones and/or royalties
2 Partnered, Alnylam-led development with US profit split and milestones/royalties ex-US
3 Partner-led with profit split
4 Partnered, Alnylam-led with profit split
Alnylam-owned, -led or -co-developed
Alnylam clinical development pipeline as of October 2026
Additional Information
Clinical Trials
Patients are at the center of everything we do and we work tirelessly to bring new medicines to those with unmet needs. Learn more about Alnylam-sponsored clinical trials and find one near you.
Capella
Learn more about the latest in Alnylam news from Capella—the destination for updates on our progress in translating the science of RNAi to innovative potential medicines.
Investors
Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Discover more information for investors.
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